Ara-C

Phase 2Small molecule

KKaryopharm Therapeutics Inc

Ara-C is one of 69 small molecule programs targeting Stem Cell Transplant. It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Stem Cell TransplantTerminated

Relapsed/refractoryRecruiting

Stem Cell TransplantPreclinical

Terminated

Relapsed/refractoryPhase 2

Recruiting

Competition

Competitive Landscape

64 competitors in Relapsed/refractory

#DrugCompanyPhaseTrials

Decadron

AAspen Global Incorporated

Phase 3

9 2

Rituxan

Roche

Phase 2

9 3

Venclexta

AbbVie

Phase 2

8 4

Fludara

Sanofi

Phase 2

7 5

Opdivo

Bristol-Myers Squibb

Phase 2

4 6

Keytruda

Merck & Co.

Phase 2

4 7

Adcetris

Pfizer

Phase 2

2 8

Gemzar

Johnson & Johnson

Phase 2

2 9

Velban

Eli Lilly

Phase 2

1 10

Eloxatin

Teva

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q2 2027

Phase 2Recruiting

Relapsed/refractory

NCT06489808

Q2 2029

PreclinicalRecruiting

Myelodysplastic Syndromes

NCT05807932

Clinical

Trials by Indication[1]

Lymphoma6 trials

Rituxan + BEAM and Auto Stem Cell Transplant for High Risk Lymphoma or Hodgkin's Disease

Completed·—·N/A·NCT01702961·75 enrolled

Trial for Patients With Newly Diagnosed Primary Central Nervous System (CNS) Lymphoma

Completed·IInternational Extranodal Lymphoma Study Group (IELSG)·Phase 2·NCT01011920·227 enrolled

Brentuximab Vedotin in Pre-transplant Induction and Consolidation for Relapsed or Refractory Hodgkin Lymphoma

Completed·GGrupo Español de Linfomas y Transplante Autólogo de Médula Ósea·Phase 1/2·NCT02243436·67 enrolled

Show 3 more trials

Efficacy of the HCVIDDOXIL Regimen in Patients With Newly Diagnosed Peripheral T-Cell Lymphoma

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT00290433·55 enrolled

Y Zevalin and BEAM in Autologous Stem Cell Transplantation (ASCT) for Lymphoma

Completed·MM.D. Anderson Cancer Center·Phase 1/2·NCT01538472·40 enrolled

Fludarabine, Mitoxantrone, and Dexamethasone (FND) Plus Rituximab for Lymphoma Patients

Completed·MM.D. Anderson Cancer Center·Phase 3·NCT00577993·210 enrolled

Acute Myeloid Leukemia4 trials

Gemtuzumab Ozogamicin and High-Dose Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Completed·AAlliance for Clinical Trials in Oncology·Phase 2·NCT00006265·60 enrolled

Study of Clofarabine & Cytosine Arabinoside Therapy for Older Adults With Acute Myeloid Leukemia

Completed·UUniversity of Alabama at Birmingham·Phase 1·NCT00081822·23 enrolled

Risk Adapted Treatment for Primary Acute Myeloid Leukemia (AML)

Completed·GGrupo Cooperativo de Estudio y Tratamiento de las Leucemias Agudas y Mielodisplasias·Phase 2·NCT01723657·862 enrolled

Show 1 more trial

Risk-adapted Therapy for Primary Acute Myeloid Leukemia

Completed·GGrupo Cooperativo de Estudio y Tratamiento de las Leucemias Agudas y Mielodisplasias·Phase 2·NCT04687098·1,034 enrolled

Leukemia4 trials

Combination Chemotherapy Plus PSC 833 Followed by Interleukin-2 in Treating Patients With Acute Myelogenous Leukemia

Completed·NNational Cancer Institute (NCI)·Phase 1/2·NCT00002925·410 enrolled

RAD001 Study in Treatment of Relapsed or Refractory Acute Lymphocytic Leukemia

Completed·MM.D. Anderson Cancer Center·Phase 1/2·NCT00968253·24 enrolled

Anti-Human CD45 Monoclonal Antibodies in Patients With Advanced Leukemia Prior to Allogeneic Stem Cell Transplantation

Completed·—·Phase 1·NCT00057005·6 enrolled

Show 1 more trial

PETHEMA-LMA10: Treatment of Acute Myeloblastic Leukemia (AML) in Patients Less Than or Equal to 65 Years

Unknown status·PPETHEMA Foundation·N/A·NCT01296178·200 enrolled

Acute Lymphoblastic Leukemia4 trials

Ponatinib With Chemotherapy for Young Adults Ph Positive Acute Lymphoblastic Leukemia

Unknown status·PPETHEMA Foundation·Phase 2·NCT02776605·30 enrolled

Study of Augmented Hyper-CVAD in Acute Lymphoblastic Leukemia Salvage

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT00890656·90 enrolled

Pethema LAL-RI/2008: Treatment for Patients With Standard Risk Acute Lymphoblastic Leukemia

Completed·PPETHEMA Foundation·Phase 4·NCT02036489·107 enrolled

Show 1 more trial

Hyper-CVAD With Liposomal Vincristine in Acute Lymphoblastic Leukemia

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT01319981·31 enrolled

Myelodysplastic Syndrome3 trials

Clofarabine Plus Cytarabine in Patients With Previously Untreated Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT00065143·60 enrolled

Azacytidine With Valproic Acid Versus Ara-C in Acute Myeloid Leukemia (AML)/ Myelodysplastic Syndrome (MDS) Patients

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT00382590·11 enrolled

Phase I/II Study of 5-Azacytidine With Ara-C in Patients With Relapsed/Refractory Acute Myelogenous Leukemia (AML) or High Risk Myelodysplastic Syndrome (MDS)

Completed·MM.D. Anderson Cancer Center·Phase 1/2·NCT00569010·36 enrolled

25 more indications

Myelodysplastic Syndromes3 trials

Gleevec/Low-Dose Ara-C Study for Elderly Patients With AML and Myelodysplastic Syndromes

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT00451997·10 enrolled

Clofarabine vs Clofarabine in Plus With Low-Dose Ara-C in Previously Untreated Patients With Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndromes (MDS).

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT00088218·95 enrolled

Phase-I/II Trial to Assess the Safety and Efficacy of Venetoclax in Addition to Sequential Conditioning With Fludarabine / Amsacrine / Ara-C (FLAMSA) + Treosulfan for Allogeneic Blood Stem Cell Transplantation in Patients With MDS, CMML or sAML (FLAMSAClax

Recruiting·HHeinrich-Heine University, Duesseldorf·Preclinical·NCT05807932·38 enrolled

Acute Promyelocytic Leukemia2 trials

Treatment of Newly Diagnosed Patients With Acute Promyelocytic Leukemia (PETHEMA LPA 2005)

Completed·PPETHEMA Foundation·Phase 4·NCT00408278·300 enrolled

Cytarabine (Ara-C) in Children With Acute Promyelocytic Leukemia (APL)

Completed·XXiaofan Zhu·Phase 4·NCT01191541·65 enrolled

Stem Cell Transplant1 trial

T-Reg Cell Kinetics, Stem Cell Transplant, REGALE

Terminated·—·N/A·NCT00578539·24 enrolled

Efficacy1 trial

Efficacy of R-CHOP vs R-CHOP/R-DHAP in Untreated MCL

Unknown status·EEuropean Mantle Cell Lymphoma Network·Phase 3·NCT00209222·360 enrolled

Stem Cell Transplantation1 trial

T-Regulatory Cell Kinetics, Stem Cell Transplantation, REGKINE

Terminated·—·N/A·NCT00578461·26 enrolled

Sorafenib1 trial

Phase I-II Study of Idarubicin, Cytarabine, and Sorafenib (BAY43-9006)

Completed·MM.D. Anderson Cancer Center·Phase 1/2·NCT00542971·78 enrolled

Safety and Efficacy1 trial

Safety and Efficacy Study of Ara-c at 18 gm/m2 Versus 12 gm/m2 for 3 Cycles Each in AML Consolidation

Unknown status·—·Phase 3·NCT01615757·180 enrolled

Chronic1 trial

PEG Interferon Alpha 2B and Low-Dose Ara-C in Early Chronic Phase CML

Completed·MM.D. Anderson Cancer Center·Phase 1·NCT00303290·76 enrolled

Memory1 trial

Cytokine-induced Memory-like NK Cells in Combination With Chemotherapy in Pediatric Patents With Refractory or Relapsed AML

Withdrawn·WWashington University School of Medicine·Phase 2·NCT04354025

Hypereosinophilic syndrome1 trial

2-Chlorodeoxyadenosine and Cytarabine in Patients With Idiopathic Hypereosinophilic Syndrome (HES)

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT00483067·13 enrolled

Relapsed/Refractory AML1 trial

Phase IIa Study Evaluating Safety and Efficacy of BL-8040 in Relapsed/Refractory AML Patients

Completed·BBioLineRx, Ltd.·Phase 2·NCT01838395·42 enrolled

Adult Acute Myeloid Leukemia1 trial

Risk-adapted Therapy for Adult Acute Myeloid Leukemia.

Completed·GGrupo Cooperativo de Estudio y Tratamiento de las Leucemias Agudas y Mielodisplasias·Phase 2·NCT01716793·354 enrolled

Older Patients1 trial

Selinexor (KPT-330) in Older Patients With Relapsed AML

Completed·KKaryopharm Therapeutics Inc·Phase 2·NCT02088541·317 enrolled

Stem Cell1 trial

T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts

Completed·—·Phase 2·NCT00368355·46 enrolled

Myeloid Leukemia1 trial

A Study for Improving the Outcome of Childhood Acute Promyeloid Leukemia

Completed·SSouth China Children's Leukemia Group·Phase 4·NCT02200978·176 enrolled

Chemotherapy1 trial

A Clinical Trail of Demethylation Drug Combined With Chemotherapy in Intermediate-risk AML

Unknown status·XXuejie Jiang·Phase 2·NCT03417427·100 enrolled

Acute Myeloblastic Leukaemia1 trial

Treatment of the Acute Myeloblastic Leukaemia in Patients Over 65 Years

Completed·PPETHEMA Foundation·Phase 4·NCT00464217·100 enrolled

Elderly Patients1 trial

Phase II Trial in Elderly Patients With AML or MDS in Complete Remission Not Eligible for Allogenic Transplant

Completed·CCiceri Fabio·Phase 2·NCT03961919·15 enrolled

Transplantation1 trial

High-dose Cytarabine/Mitoxantrone Followed by Autotransplantation for t-MDS/t-AML

Completed·UUniversity of Chicago·Phase 2·NCT00774046·32 enrolled

Lymphoid Malignancies1 trial

Natural Killer (NK) Cell Adback After Allogeneic Stem Cell Transplant With Campath-IH Plus Chemorx for Patients With Lymphoid Malignancies

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT00536978·22 enrolled

Relapsed/Refractory1 trial

Linperlisib Combined With Immunochemotherapy in Relapsed/Refractory LBCL

Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 2·NCT06489808·89 enrolled

Diffuse Large B-Cell Lymphoma1 trial

Dose Dense Chemotherapy + Rituximab +/-Intensified High Dose Chemoimmunotherapy With Support of Peripheral Autologous Stem Cell in Diffuse Large B-Cell Lymphoma

Unknown status·FFondazione Italiana Linfomi - ETS·Phase 3·NCT00499018·399 enrolled

Leptomeningeal Metastases1 trial

Concurrent Involved-field Radiotherapy and Intrathecal Chemotherapy for Leptomeningeal Metastases From Solid Tumors

Completed·TThe First Hospital of Jilin University·Phase 2·NCT03082144·53 enrolled

AML1 trial

HLA-mismatched MST vs HLA-matched NST for AML in Intermediate-risk

Completed·TThe Affiliated Hospital of the Chinese Academy of Military Medical Sciences·Phase 3·NCT02461121·156 enrolled

Multiple Myeloma1 trial

UARK 98-026 TT II: Multiple Myeloma Evaluating Anti-Angiogenesis With Thalidomide and Post-Transplant Consolidation Chemotherapy

Completed·UUniversity of Arkansas·Phase 3·NCT00083551·668 enrolled

Sources

References

[1]
ClinicalTrials.gov — 49 trial records for Ara-C. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Ara-C compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Ara-C. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Karyopharm Therapeutics Inc company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: KKaryopharm Therapeutics Inc

Ara-C

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q2 2027

Q2 2029

Trials by Indication[1]

Lymphoma6 trials

Acute Myeloid Leukemia4 trials

Leukemia4 trials

Acute Lymphoblastic Leukemia4 trials

Myelodysplastic Syndrome3 trials

Myelodysplastic Syndromes3 trials

Acute Promyelocytic Leukemia2 trials

Stem Cell Transplant1 trial

Efficacy1 trial

Stem Cell Transplantation1 trial

Sorafenib1 trial

Safety and Efficacy1 trial

Chronic1 trial

Memory1 trial

Hypereosinophilic syndrome1 trial

Relapsed/Refractory AML1 trial

Adult Acute Myeloid Leukemia1 trial

Older Patients1 trial

Stem Cell1 trial

Myeloid Leukemia1 trial

Chemotherapy1 trial

Acute Myeloblastic Leukaemia1 trial

Elderly Patients1 trial

Transplantation1 trial

Lymphoid Malignancies1 trial

Relapsed/Refractory1 trial

Diffuse Large B-Cell Lymphoma1 trial

Leptomeningeal Metastases1 trial

AML1 trial

Multiple Myeloma1 trial

References

Drug Info

Quick Facts

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